Following on from yesterday’s announcement about the restarting of our research function, the BDFA is pleased to announce our collaboration with a group of families who have children with CLN2 and are accessing enzyme replacement therapy (ERT).
This campaign is looking to raise funds toward a potential compassionate use programme for loss of eyesight in children affected by CLN2 who are accessing Brineura © The hope is it will treatment options to these families and future children who are diagnosed with CLN2 and whose families choose ERT.
“The families have been working tirelessly preparing for this launch and we are very proud to be working together to make a difference to the Batten Disease Community, for children of today and the future,” says a parent of a child accessing Brineura ©.
The campaign launches on Just Giving today. If for any reason the Brineura© in the eyes programme does not go ahead or if funds raised are not all needed, the money will be designated for other research and supporting families in our community.
We can only make this treatment a reality with your help so if you think you can help in any way, we would love to hear from you.
If you have an event you would like to set up, you can set up your fundraising page through the Just Giving link which is LIVE now and ready to accept donations.
Thank you in advance for your support, it is greatly appreciated by us all and we look forward to sharing updates with you as the campaign progresses!
The BDFA exists to represent the entire community touched in anyway by Batten disease and we want as many people as possible to be involved throughout the community. We hope we can count on your support.